Pursuing Next Generation Nucleic Acid Therapies

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Healthcare

Published

2022

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#1#2Cautionary Note Regarding Forward Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipate," "expect," "plan," "may," "will," "continue," and similar expressions (as well as other words or expressions referencing future events or circumstances) are intended to identify forward-looking statements. All statements other than statements of historical fact in this presentation are forward looking including, but not limited to, statements regarding of Exicure, Inc. ("Exicure" or the "Company"); including the development of product candidates based on its proprietary SNA TM technology; the initiation, timing, progress, scope and results of the Company's preclinical studies, clinical trials and research and development programs; the timing, availability and presentation of preclinical, clinical and regulatory developments; the potential benefits of the Company's product candidates; the development and the commercial potential, growth potential and market opportunity for the Company's product candidates, if approved, and the drivers, timing, impact and results thereof; potential and future results of current and planned collaborations; the proposed benefits of any of the Company's partnered programs; and the timing or likelihood of regulatory filings and approvals. Forward-looking statements are based on management's current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: market and other conditions; the risk that the ongoing COVID-19 pandemic and the associated containment efforts may disrupt the Company's business and/or the global health care system (including its supply chain) more severely than it has to data or more severely than anticipated; the Company's preclinical or clinical programs do not advance or result in approved products on a timely or cost effective basis or at all; the results of early clinical trials are not always being predictive of future results; unexpected safety or efficacy data observed during preclinical or clinical studies; the failure of the data from the Company's preclinical trials to be indicative in human trials; clinical site activation rates or clinical trial enrollment rates that are lower than expected; the cost, timing and results of clinical trials; changes in the regulatory environment; exposure to litigation or other disputes; the ability of the Company to protect its intellectual property rights; the ability of the Company to obtain or maintain collaborations and/or collaborate successfully with strategic partners; the actual funding required to develop and commercialize the Company's product candidates and operate the Company and the impact of the completion of the Company's previously reported internal investigation on the Company's business and diversion of management time and attention on related issues, including any related investigations or proceedings, shareholder lawsuits, reputational harm, or the possibility that executives or other employees may resign. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in the Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 16, 2022, as well as discussions of potential risks, uncertainties, and other important factors in its subsequent filings with the Securities and Exchange Commission. All information in this presentation speak only as of the date of this presentation, and the Company undertakes no obligation to update this information or to publicly announce the results of any revisions to any of such statements to reflect future events or developments, unless required by law. exicure © 2022 Exicure, Inc. All Rights Reserved. | 2#3#4#5SNAS: Next Generation Nucleotide Delivery Platform exicure#6#7#8Three Drivers of Superior Efficacy in Neuroscience 1 Higher Cell Uptake, Leading to Higher Potency at Same Dose SNA Linear OLIGO DAPI OLIGO DAPI Superior cell uptake¹ vs. linear oligonucleotide with SNA detection observed across multiple cell types Broad CNS Penetration and Uptake in all Neuronal Cells Nucleus (hematoxylin) 2 anti-MALAT1 ON probe Deep Cerebellum In Situ Hybridization of SNA in the Brain (Rat) via ICM dosing SNAS² reach deep brain regions and achieve uptake in neurons, microglia and astrocytes 3 Higher Persistence in the CNS, Leading to Prolonged Efficacy Nusinersen Alone SNA- Nusinersen Radiolabeled biodistribution shows SNAs³ stay longer in the CNS compared to linear nucleotide therapeutic 1) In vitro comparison in cortical neurons testing MALAT1 knockdown with IC 50 for SNA (141 nM) and linear oligonucleotide (464 nM), 2) In vivo testing with MALAT1 knockdown via intracisterna magna (ICM) route of administration 3) In vivo comparison of rats dosed intrathecally with comparison shown at seven days after one-time administration. exicure © 2022 Exicure, Inc. All Rights Reserved. | 8#9#10#11#12#13#14#15#16#17#18#19#20#21#22#23

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